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2014 | 1 |
2021 | 1 |
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Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.
Nat Commun. 2021 Jul 12;12(1):4251. doi: 10.1038/s41467-021-24524-8.
Nat Commun. 2021.
PMID: 34253733
Free PMC article.
Clinical Trial.
SAFETY AND TOLERABILITY OF MRI-GUIDED INFUSION OF AAV2-hAADC INTO THE MID-BRAIN OF NON-HUMAN PRIMATE.
San Sebastian W, Kells AP, Bringas J, Samaranch L, Hadaczek P, Ciesielska A, Macayan M, Pivirotto PJ, Forsayeth J, Osborne S, Wright JF, Green F, Heller G, Bankiewicz KS.
San Sebastian W, et al.
Mol Ther Methods Clin Dev. 2014 Oct 15;3:14049-. doi: 10.1038/mtm.2014.49.
Mol Ther Methods Clin Dev. 2014.
PMID: 25541617
Free PMC article.
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